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Early Onset, But Late Diagnosis of a Rare Disease
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Abstract [+]
One of the most common sphingolipidosis, Gaucher disease (GD) remains rare till date. A case report of a 56-year-old patient diagnosed with GD is presented herein. Her sister was known to have Gaucher disease. Her personal medical record consisted of splenectomy, anemia, recurrent infections, and bone lesions at a young age. Taking into consideration her personal and familial history, the clinical and paraclinical examinations, she was diagnosed with Gaucher disease which was confirmed with enzyme and gene testing. Upon introduction of specific enzyme replacement treatment for Gaucher patients,much evidence demonstrated the substantial improvement of hematological and visceral parameters. However, it has been observed that the bone tissue does not respond equally to the treatment.
Learning Points
• The physician should always investigate the splenomegaly of unknown etiology before deciding to do a splenectomy
• Bone lesions in Gaucher disease are sometimes irreversible, hence the importance of early diagnosis of this rare disease
Keywords
Gaucher disease; Bone lesion; Splenomeagly.
Acronyms
GD: Gaucher disease; Hb: Hemoglobin; MRI: Magnetic resonance imaging; ERT: Enzyme replacement therapy.
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Efficacy and Safety of Clarithromycin, Lenalidomide and Dexamethasone (Bird) Therapy for Japanese Patients with Relapsed or Refractory Multiple Myeloma
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Abstract [+]
Object
It is difficult for the elderly, those with complications, and those who live in remote areas to visit the hospital, and as a result, there are limits on the drugs they are able to use. It is therefore effective to prescribe such patients oral medications that have few adverse effects and in regimens that require few hospital visits. Clarithromycin can induce cell death by autophagy and it has a direct antitumor effect. There have been reports of the outcomes of Lenalidomide and Dexamethasone therapy with Clarithromycin which is administered orally and is safe on multiple myeloma. However, in Japan, there have been few studies. Here, we report on Clarithromycin, Lenalidomide and Dexamethasone therapy in our hospital.
Method
We analyzed 7 patients with relapsed refractory or refractory multiple myeloma who were treated at this hospital between January 2012 and December 2014. The Clarithromycin, Lenalidomide and Dexamethasone therapy were administered in a 28-day cycle as follows: Clarithromycin 400 mg/day for 28-days, Lenalidomide 15 mg/day for 21-days, and Dexamethasone was administered in a dose of 20 mg once per week. The response criteria used were standard International Myeloma Working Group (IMWG) Uniform Response Criteria. and adverse events were graded according to the national cancer institute-common terminology criteria for adverse events (NCI-CTCAE) Ver. 4. Statistical analysis was performed using Easy R (EZR).
Result
The response to Clarithromycin, Lenalidomide and Dexamethasone therapy were selective catalytic reduction (sCR) in 2 patients, CR in 1 patient, per rectum (PR) in 3 patients, and standard deviation (SD) in 1 patient. Response rates of PR or better were observed in 86% of the patients. Duration of response was median 316-days (range, 160-522-days). Median oculus sinister (OS) period was 1,907 days. Median OS following discontinuation of the study was 1,385 days. Hematological adverse events were G1-2 anemia in 3 patients and G3-4 anemia in 1 patient. G1-2 thrombocytopenia was observed in 1 patient and G3-4 thrombocytopenia was observed in 1 patient. Leukopenia of G1-2 was observed in 6 patients but G3 was not observed. Non-hematological adverse events were G1-2 liver disorder in 6 patients, G1-2 skin rash in 3 patients, and G1-2 constipation in 2 patients. G4 adverse events were fainting and duodenal ulcer in 1 patient each.
Conclusion
Clarithromycin, Lenalidomide and Dexamethasone can be safely and effectively administered in the relapsed refractory multiple myeloma
Keywords
Multiple myeloma; Bird; Clarithromycin, lethal dose (Ld).
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Editor-in-Chief
Hongyu Wang, MD
Chief Physician Department of vascular Medicine Peking University Shougang Hospital NO.9 Jinyuanzhuang Road Shijingshan District, 100144, Beijing China
Associate Editors
Arnon Blum, MD
Director of Medicine Professor Department of Medicine Baruch Padeh Poria Medical Center affiliated to the Faculty of Medicine Bar-Ilan University Ramat Gan, 5290002, Israel